Evaluation of Lyso-Gb1 as a biomarker for Gaucher disease treatment outcomes using data from the Gaucher Outcome Survey

Abstract Background Patients with Gaucher disease (GD) require continual monitoring; however, lack of specific disease biomarkers was a significant challenge in the past. Glucosylsphingosine (lyso-Gb1) has been shown to be a reliable, key, specific, and sensitive biomarker for diagnosis, prognosis,...

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Detalhes bibliográficos
Main Authors: Ari Zimran, Shoshana Revel-Vilk, Tama Dinur, Majdolen Istaiti, Jaco Botha, Elena Lukina, Pilar Giraldo, Patrick Deegan, Stephan vom Dahl
Formato: Artigo
Idioma:Inglês
Publicado em: BMC 2025-01-01
Colecção:Orphanet Journal of Rare Diseases
Assuntos:
Lyso-Gb1
Glucosylsphingosine
Gaucher disease
Biomarker
Acesso em linha:https://doi.org/10.1186/s13023-024-03444-y
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